The interventional cardiologist and professor, Duke University School of Medicine, discussed other populations that may benefit from XC001 and the challenges and opportunities with a surgical delivery of treatment.
There was a continued trend toward improvement of overall survival with omidubicel at 73% compared with UCBT at 60%.
Foundation Fighting Blindness is helping to support the development of gene therapies for retinal diseases.
The chief executive officer of CG Oncology discussed the role of CG0070 in patients who are unresponsive and responsive to Bacillus Calmette-Guerin immunotherapy.
Takashi Kei Kishimoto, PhD, and Carsten Brunn, PhD, the chief science and executive officers of Selecta Biosciences, discussed the immunogenicity-mitigating properties of ImmTOR.
Dolores Schendel, PhD, chief executive and chief scientific officer, Medigene, discussed the company’s collaboration with 2seventy bio to develop TCR T-cell therapies.
The chief scientific officer of cell therapy at Poseida Therapeutics discussed the company’s CAR-T and TCR technologies.
The director of the Mellen Center for MS Treatment and Research at Cleveland Clinic discussed upcoming new therapies for relapsing MS.
The chief medical officer of QurAlis discussed the antisense oligonucleotide therapy QRL-201.
The director of the Clinical Myeloma Program at NYU Langone Health’s Perlmutter Cancer Center discusses the next steps for CAR T-cell therapy in the treatment of patients with relapsed/refractory multiple myeloma.
The medical director of the UCLA Bone Marrow Transplantation Stem Cell Processing Center discussed results from a phase 1b trial for allo-HCT alternative Orca-T that he presented at Tandem’s 2024 conference.
The professor and director of the Myeloma Center at the University of Arkansas for Medical Sciences discussed the clinical implications of CAR T-cell therapy for patients with relapsed/refractory multiple myeloma.
Experts shared opinions about the future of gene therapy in sickle cell and other rare diseases.
A discussion of the course of gene therapy compared with stem cell therapy.
A discussion of the course of gene therapy compared with stem cell therapy.
A discussion of the course of gene therapy compared with stem cell therapy.
The senior investigator from NINDS discussed findings investigating serious AEs across different trials.
The professor of ophthalmology from Radboud University, The Netherlands, discussed ongoing gene therapy trials in Stargardt disease.
The assistant professor at Mayo Clinic School of Medicine discussed plans for further research and a phase 2/3 study.
Krystal Biotech's investigational topical gene therapy has shown promise in DEB. It is designed to deliver COL7A1 and restore C7 protein in patients with the rare disorder.
The assistant member of the bone marrow transplantation and cellular therapy department at St. Jude Children’s Hospital discussed preclinical research presented at ASH 2022.
Michael Parini, chief executive officer and director, Freeline Therapeutics, discussed the company’s pipeline and mission.
The physician from St. Jude Children’s Research Hospital discussed the importance of involvement from patients and families while researching the long-term impact of CAR-T.
The executive director of Hemophilia Foundation Southern California discussed issues with minorities accessing care for rare diseases and strategies to mitigate them.
The associate professor from Perelman School of Medicine, University of Pennsylvania, discussed co-administered CART22-65s and huCART19 in relapsed/refractory acute lymphoblastic leukemia.
The clinical associate professor from the Rogel Cancer Center at Michigan Medicine discussed the future of CAR T-cell therapy in mantle cell lymphoma patients.
The president and head of R&D at Taysha Gene Therapies discussed his expectation that younger patients my see even better results with the company’s gene therapy than the adult patients treated so far.
The associate professor of medicine at Sylvester Comprehensive Cancer Center discussed updated data from the phase 3 ALLELE study.
The associate professor at the University of Washington, and principal investigator at Seattle Children's Research Institute discussed results from a leukemia mouse model.
The professor of medicine at Duke Cancer Institute discussed uptake and research with the cord blood therapy since its approval.